The daily disease management burden placed on Type 1 diabetics means scientists have long sought a way to fix the root cause of the disease. A treatment that lessens the need to monitor blood sugar levels would improve the lives of the 3 million Americans with the disease--and disrupt the diabetes market.
Decades of attempts have fallen short though, with treatments either failing to stop the decline of insulin-releasing cells, or putting patients at risk by shutting off other bits of the immune system too. Data from a clinical trial of 80 Type 1 diabetics suggests that DNA vaccines could avoid these problems. Writing in Science Translational Medicine, researchers report that 12 weekly shots may help preserve the remaining pancreatic insulin-producing beta cells without causing serious side effects. Advances in the understanding of genetics underpin the research.
"The idea here is to turn off just the rogue immune cells that are attacking the pancreas and killing the beta cells that secrete insulin," Stanford University professor Dr. Lawrence Steinman told Reuters. To perform this task Steinman and collaborators at Stanford and Leiden University Medical Center in the Netherlands created a genetically engineered plasmid. The DNA vaccine targets a precursor protein in the blood called proinsulin. "This effectively triggers an off-switch," Steinman said.
Having been reassured by the safety profile of the vaccine, Steinman and his collaborators now plan to recruit up to 200 patients for a longer clinical trial. The researchers hope to see evidence that the vaccine can slow or stop disease progression in younger patients who still have plenty of insulin-releasing cells. This would lessen the need to inject insulin as a replacement therapy.
Steinman has co-founded a biotech, called Tolerion, to commercialize the vaccine. Bloomberg reports that the startup is looking at partnerships and "other vehicles for moving ahead" with the vaccine. Eli Lilly ($LLY), Novo Nordisk ($NVO), Sanofi ($SNY) and other Big Pharma companies currently compete for market share in diabetes. Success in treating Type 1 diabetes could see Tolerion apply the approach to other illnesses that stem from an immune malfunction, such as Grave's disease.